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How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 proteinexpression in muscles in these patients.
A team led by researchers at Baylor College of Medicine and the Czech Academy of Sciences has uncovered a new piece of the puzzle of how geneexpression is orchestrated. Published in the journal Science, the findings reveal a novel mechanism that coordinates the assembly of components inside cells that control geneexpression.
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The exact mechanism depends on the disease in question.
Scientific evidence shows how the cognitive decline in Alzheimer's disease (AD) is caused by the buildup of amyloid beta proteins, which promote synaptic malfunction.
Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.
Crop legumes are an integral part of sustainable agriculture, as several of these species represent an important protein source for both human and animal populations. Legumes engage in a unique and beneficial interaction with a group of soil bacteria, collectively called rhizobia.
The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Protein that manipulates geneexpression inside the cell nucleus could inspire new RSV drugs, vaccines.Protein that manipulates geneexpression inside the cell nucleus could inspire new RSV … Continue reading →
The function of a protein can depend on its abundance in a cell. So, when investigating the properties of a new protein, it is essential to make sure that the same amount is produced by every cell. Researchers at Baylor College of Medicine and Rice University have found a new way to do just that […].
Professor Norikazu Ichihashi and his colleagues at the University of Tokyo have successfully induced geneexpression from a DNA, characteristic of all life, and evolution through continuous replication extracellularly using cell-free materials alone, such as nucleic acids and proteins for the first time.
Brazilian research group shows that valproic acid (VPA), used to treat epilepsy since the 1960s, modulates geneexpression in tumor gene models and acts on DNA conformation and the histones in chromatin Results of recent studies involving valproic acid, used for decades as an anti-convulsant drug, show that it can interact with the conformation of (..)
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Study highlights role of disordered protein interactions in gene expression.A
By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.
By tying these to underlying disease-protein-pathway relationships, the AI was also able to suggest a list of drugs – many of them untested in COVID-19 – that could be candidates for treatments of these complications.
Mount Sinai researchers have discovered that Polycomb complexes, groups of proteins that maintain geneexpression patterns, are essential for proper skin development, according to a paper published in Genes & Development on February 18.
The detailed complexity of these processes can be captured by creating models that combine correlates of gene and proteinexpression, providing insight into the molecular composition of tissues. The barcode sequence is followed by a unique molecular identifier, which enables accurate digital counting of gene transcripts.
Source : Biology Textbooks Wrong? New Research Reveals the Secret Behind a Key Cellular Process (scitechdaily.com) New research has identified and described a cellular process that, despite what textbooks say, has remained elusive to scientists until now — precisely how … Continue reading →
A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates geneexpression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.
Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting geneexpression. Thus, targeting menin may be an effective strategy for patients with high unmet needs.
bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. He added: “We have seen no significant gene misregulation attributable to the insertion event.
Capturing how RNA polymerase enzymes kick off transcription On scales too small for our eyes to see, the business of life happens through the making of proteins, which impart to our cells both structure and function.
Study shows that tumor-suppressor protein p53 brings speckles and DNA together to boost geneexpression PHILADELPHIA – A team led by scientists at the Perelman School of Medicine at the University of Pennsylvania has illuminated the functions of mysterious structures in cells called “nuclear speckles,” showing that they can work in (..)
Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling geneexpression.”.
Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.
For the first time, researchers describe how Rho protein really stops geneexpression COLUMBUS, Ohio – New research has identified and described a cellular process that, despite what textbooks say, has remained elusive to scientists until now – precisely how the copying of genetic material that, once started, is properly turned off.
Findings suggest one factor that may contribute to disparities in COVID-19 infection New York – In a study published in JAMA today, Mount Sinai researchers report findings that shed some light on the disproportionate impact of COVID-19 on Blacks, who have experienced rates of infection and death that are much greater, in some areas twice […]. (..)
Innovation S-curve for the pharmaceutical industry Transcription factors for genetically modified cells is a key innovation area in the pharmaceutical industry Transcription factors are proteins which control geneexpression by controlling the rate of transcription of genetic information from DNA to RNA.
The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Enabling cells to talk to computers.Genetically encoded reporter proteins have been a mainstay of biotechnology research, allowing scientists to track geneexpression, understand intracellular processes and debug … Continue (..)
The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. A gene contains instructions within our DNA.
AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases.
UNC researchers now show that these two factors can directly associate with one another, modulating cancer-cell-specific programs of geneexpression. ” They found that EZH2 possesses two different binding patterns on chromatin in acute leukemia cells, eliciting two distinct gene-regulatory programs (Figure 1).
There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy. Secondly, other funded research is exploring differential geneexpression regulated by DNA methylation.
This innovative technology, capable of carrying large protein payloads, has demonstrated potential in restoring muscular function in a mouse model of Duchenne muscular dystrophy (DMD) through the delivery of a full-length dystrophin protein.
Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate geneexpression without altering the sequence or structure of DNA.
This finding stemmed from more than two decades of her basic research into a gene in pancreatic islets called TXNIP. Fifty-three proteins showed significantly altered relative abundance over time in response to verapamil. Fifty-three proteins showed significantly altered relative abundance over time in response to verapamil.
Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. Thus, restoring the presence of this particular gene can help to restore several of the functions of the brain.
The drug, UCB0107, is designed to impede or decrease the buildup of tau proteins in the brain, which leads to nerve cell damage and death. Omega’s platform is designed to adjust geneexpression to healthy levels rather than switching genes on and off.
Beta catenin meanwhile is a signalling protein in the Wnt pathway – in particular a downstream transcription factor (TCF) implicated in a range of solid tumour types including colorectal, liver, breast, prostate, uterus and lung cancers.
Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. This can be attributed to the fact that the production of biopharmaceuticals requires living expression systems.
Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence geneexpression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.
Hybridization Tests – These tests utilize complementary DNA or RNA probes to detect and quantify specific genetic targets, enabling the identification of pathogens, genetic variations, and geneexpression patterns. Such test can be used for geneexpression profiling, genotyping and detecting chromosomal abnormalities.
Abell Chair in Molecular and Cellular Biology – and Pengbo Cao, a postdoctoral researcher in Whiteley’s lab, discovered a gene that drives the switch. By measuring bacterial geneexpression in human tissue samples, the researchers identified a biomarker for the transition. and Nelson D.
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